histiocytic neoplasms
Conditions
Interventions
Experimental group:Treatment of FCN - 159
Sponsors
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
16 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1.> 16 years of age. 2.Patients with histologically confirmed LCH, ECD or other histiocytic tumors. 3.Ability to provide sufficient numbers of histopathological sections or tumor tissue samples for central pathology review and central laboratory testing for the following biomarkers: ERBB3, RAF-1, BRAF, ARAF, HRAS, KRAS, NRAS, MEK (MAP2K1 and MAP2K2) and other MEK upstream genes. 4.Patients with LCH require lesions involving multiple systems (more than one system) or multiple lesions involving a single system (more than one lesion). 5.Presence of evaluable lesions based on PET response criteria (PRC) is required. 6.Treatment-na?ve or relapsed or refractory adult patients with LCH, ECD or other histiocytic tumors can be included. 7.Expected survival is at least 3 months. 8.The ECOG score is 0-2 points. 9.Participants are able to understand and voluntarily sign a written informed consent form. 10.Negative serum human chorionic gonadotropin (HCG) pregnancy test in premenopausal women within 7 days prior to initiating dosing. Serum human chorionic gonadotropin (HCG) pregnancy test can be omitted for surgically infertile or infertile women >= 1 year postmenopausal. 11.For participants of childbearing potential: during treatment and for at least 90 days after the last dose, participants must agree to use a highly effective method of contraception (ie, double-barrier contraception, condoms, oral or injectable contraceptives, IUDs, and other contraceptive methods). Male participants must agree to avoid sperm donation for at least 90 days after the last treatment. 12.Adequate bone marrow function: in the absence of blood transfusion, blood products, and granulocyte colony-stimulating factor, the absolute value of neutrophils is >= 1.5 x 10^9/L, hemoglobin is >= 90 g/L, and platelets are >= 100 x 10^9/L. Patients whose cytopenias due to the disease itself are lower than these thresholds can be exempted and enrolled according to the investigator's comprehensive judgment of the disease. 13.Sufficient liver and kidney function: Aspartate aminotransferase (AST), Alanine aminotransferase (ALT) 1.5 x ULN, creatinine clearance rate (CCr) >= 50 mL/min (based on Cockcroft-Gault formula); Albumin>=3g/dL. 14.Coagulation function: International normalized ratio (INR) and activated partial thromboplastin time (APTT) <= 1.5ULN.
Exclusion criteria
Exclusion criteria: Participants who meet any of the following conditions shall not be included in this clinical study: 1. Participants who have previously received one of the following: (1) Received chemotherapy, targeted therapy, immunotherapy, biological therapy, experimental therapy or herbal anti-tumor therapy for histiocytic tumors within 4 weeks or 160/100 mmHg. 4. Participants with dysphagia, active digestive diseases, malabsorption syndrome, or other conditions that might affect the absorption of the study drug. 5. Participants with previous or current retinal vein occlusion (RVO), retinal pigment epithelial detachments (RPED), glaucoma or other significant abnormality in ophthalmic examination. 6. Interstitial pneumonia, including existing clinically significant radiation pneumonitis. (Interstitial pneumonia caused by primary lung involvement is not included.) 7. Cardiac dysfunction or concomitant diseases meeting any one of the following conditions will be excluded: (1) Three 12-lead electrocardiogram (ECG) measurements performed at the study site during the screening period for which the mean value of three measurements was calculated according to the QTcF formula using the instrument, with QTcF > 470 ms; Participants with risk factors for QTcF prolongation, such as uncorrectable hypokalemia, hereditary long QT syndrome; or receiving drugs that prolong QTcF interval (mainly class Ia, Ic, III antiarrhythmic drugs). Drugs with potential to prolong QTcF interval. (2) New York Heart Association (NYHA) Class >= 3 congestive heart failure; (3) Clinically significant arrhythmia, including but not limited
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective response rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| disease control rate;time to response;progression-free survival;overall survival time; | — |
Countries
China
Contacts
Public ContactJian Li
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Outcome results
None listed